Gene Transfer in Brain Disorders
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Neurobiology".
Deadline for manuscript submissions: 15 June 2024 | Viewed by 199
Special Issue Editors
Interests: gene transfer/therapy; transgenic mice; hippocampus rejuvenation; brain disorders; intellectual ability and disability
Special Issues, Collections and Topics in MDPI journals
Interests: gene therapy; AAV vectors; Alzheimer's disease; Parkinson's disease; Niemann-Pick disease
Interests: psychiatry; behavioral health; dentate granular neurons; mindfulness based intervention; intensive care unit; depression; social media; mood; anxiety; stress
Special Issue Information
Dear Colleagues,
Of all the organs in the body, the brain—protected by the endothelial blood–brain barrier (BBB), which regulates the internal milieu—is the most complicated in terms of the numbers of structures/functions involved, and the numbers of gene transcripts that change over time. The pathogenesis of the brain can be viewed via the lens of the dysregulation of gene variants expressions that are reported, not only in intellectual disabilities (i.e., Down syndrome, Rett syndrome, DiGeorge syndrome, Fragile X syndrome, autism spectrum disorders), and age-regulated neurodegenerative diseases, (i.e., Alzheimer, Huntington, Parkinson’s diseases), but also in the etiology of epilepsy, brain trauma, brain cancer, Creutzfeldt–Jakob diseases, and other complex psychiatric diseases (schizophrenia, bipolar disorder, substance use disorders). Partially due to the BBB, most of the pharmacological treatments have limited efficacy, whereas surgical or neuromodulation interventions have limited indications. In contrast, gene transfer may specifically target and correct the underlying mechanisms of a disease. Various viral vector types (including retrovirus vectors, lentivirus vectors, adenovirus vectors, herpes simplex virus type 1) have been recently used for phase I/II clinical trials. Adeno-associated virus (AAV) vectors, with their well-defined in vivo safety profile, have emerged as one of the most important vectors for the treatment of brain disorders, including the delivery of CRISPR-Cas9 genome-editing machinery.
Dr. Tam Thanh Quach
Dr. Lluís Samaranch
Dr. Anne-Marie Duchemin
Guest Editors
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Keywords
- intellectual disability
- neurodegenerative disorders
- psychiatric diseases
- gene transfer
- AAV
